Lumena Raises $23 Million to Advance Drug for Rare Liver Diseases

San Diego-based Lumena Pharmaceuticals says today it has raised $23 million in a Series A round that is intended to carry the company through mid-stage trials of its lead drug candidate for treating a rare group of metabolic disorders that cause bile acid to build up in the liver.

Advancing the drug, LUM001, as a potential treatment for cholestatic liver diseases has been something of a case study in perseverance, according to Mike Grey of Pappas Ventures. Grey has been serving as the company’s CEO since Lumena was founded in 2011. He previously headed San Diego’s Auspex Pharmaceuticals, SGX Pharmaceuticals, and Trega Biosciences.

Lumena in-licensed LUM001 from Pfizer, which had conducted extensive clinical trails of the drug for its potential as a cholesterol-lowering compound, Grey told me by phone yesterday. More than 1,400 people had participated in 12 clinical trials before Pfizer decided to halt development.

But Grey said the drug had a champion in John McKearn, a managing director at RiverVest Venture Partners. McKearn remembered the compound from his years as head of discovery research at Pharmacia. Pfizer acquired the Swedish drug maker in 2002. The RiverVest director co-founded Lumena with Grey to revive development of the drug, and the two VC firms provided seed funding.

Alta Partners joined with RiverVest and Pappas to provide funding for today’s Series A round.

LUM001 is being developed as a possible therapy for cholestatic liver diseases, which cause bile acids to build up in the liver and in blood serum, leading to progressive liver damage and physical symptoms that include an intense and insatiable itch. Current treatment typically requires a surgical procedure that diverts bile into an ostomy bag. The surgery can lower bile acid levels and improve liver function, but at some cost to patients’ quality of life.

Bile acids are chemicals that are made by the liver from cholesterol. They help to absorb fats and fat-soluble vitamins. Most of the bile acids secreted by the liver are reabsorbed in the last portion of the small intestine, but choleastic liver diseases prevent bile acids from being reabsorbed.

Lumena says clinical studies have shown that LUM001 can reduce serum bile acid levels and may be effective in managing symptoms in many patients with cholestatic liver diseases. The company plans to begin a mid-stage trial of the drug in adults with Primary Biliary Cirrhosis, which causes inflammation and damage of the bile ducts in the liver. The company also plans to initiate mid-stage studies of LUM001 in children with rare congenital types of cholestatic liver disease known as Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis.

In a statement from the company, RiverVest’s McKearn says, “LUM001 could dramatically impact patient health for a population in desperate need of more effective treatment options, and we are glad to be a part of this potentially game-changing company.”